But regulatory flexibility alone cannot guarantee patient access. Beach emphasises that health technology assessment, reimbursement, NHS readiness, and supply considerations must be connected much earlier in development, rather than addressed sequentially after approval.

"The system needs to work together from the start, not sequentially at the end," Beach said.

That coordination must also extend internationally, with greater alignment on evidence expectations and opportunities to reuse data across jurisdictions while preserving independent regulatory decision-making.

Hollowood sees a similar challenge. Innovative solutions may be accepted for one disease or one company, but the lesson often stays there.

That becomes increasingly difficult to justify as advanced therapies become platform technologies.

Once researchers have accumulated evidence around a particular vector, delivery route, toxicology profile, or manufacturing approach, should every subsequent programme have to recreate all of that knowledge from the beginning?

Hollowood argues that greater ability to leverage evidence across programmes – and potentially across companies – could eliminate duplication while maintaining appropriate safeguards.

The cumulative effect could be substantial.

Reducing unnecessary development costs, shortening timelines, improving the probability of technical and regulatory success, and providing greater visibility into reimbursement all change the investment equation.

"There are tens of programmes that have been across my desk that would flip to fundable straight away," Hollowood said.

Not every rare disease programme will suddenly become viable. Hollowood is clear about that. But the inability to solve every constraint should not prevent the ecosystem from addressing the ones it can.

Each improvement can move the line.

Too often, she argues, patients enter the conversation as recipients of a finished therapy – or as people developers need to find when it is time to enrol a clinical trial.

Her experience has been almost the reverse.

Faced with a promising therapy that had no conventional route forward, patients and families became fundraisers, research supporters, regulatory advocates, and development partners. Cerolini's organisation has implemented new financing approaches combining venture philanthropy with traditional investment and worked across sectors to find a viable route into the clinic.

At one point during the conversation, she described the circumstances plainly: patient organisations helping fund gene therapy clinical development through grassroots fundraising, including bake sales and marathon runners.

It is an extraordinary response to a structural problem, but Cerolini does not see patient involvement simply as a mechanism for filling funding gaps.

Patients can convene stakeholders who otherwise operate in separate parts of the system. They bring lived experience and a sense of urgency that is difficult to reproduce institutionally. Moreover, because they encounter the entire pathway, rather than one individual piece of it, they can sometimes see connections that individual organisations cannot.

"I think that's a secret weapon right now," Cerolini said.

That requires moving beyond the language of patient centricity toward something more consequential: patients as partners in designing the pathway itself.

"We really need to rethink about the patient as the centre, rather than thinking about, 'OK, when the therapy comes, here is where we need to find the patients,'" she said.

Hollowood sees tremendous goodwill across patient organisations, industry, investors, regulators, governments, and reimbursement bodies. The difficulty is that each participant operates within different constraints and incentives, while no single participant controls the entire pathway.

That makes alignment as important as innovation.

And the stakes extend beyond the rare disease community.

As medicine becomes increasingly precise, therapies are being developed for populations defined not simply by a disease, but by specific genetic and biological characteristics. Now, the questions rare disease developers are confronting – how to generate evidence in small populations, finance highly targeted therapies, use platform knowledge efficiently, and create appropriate regulatory and reimbursement pathways – may increasingly become questions for medicine as a whole.

"This is well beyond rare diseases," Cerolini said. "It's how we are turning scientific progress into benefits for humankind."

Today, the vast majority of rare diseases still have no available treatment. Cerolini's ambition for the next five years is deliberately bold: begin reversing that equation.

Doing so will require continued breakthroughs in laboratories. But it will also require innovation in places that may receive far less attention – clinical development, manufacturing, financing, regulation, reimbursement, and collaboration.

Because, increasingly, the focus is shifting from whether the science can produce a therapy, to whether the system around that science can find a way to deliver it.

Cerolini, Hollowood, and Beach will continue this conversation at the DIA–MHRA Global Summit in London during the Day 2 opening session, Advanced Therapies for Rare Diseases, examining how new approaches across the ecosystem can help translate scientific progress into meaningful outcomes for patients.